EP3612191B1 - Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie - Google Patents
Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophieInfo
- Publication number
- EP3612191B1 EP3612191B1 EP18788380.6A EP18788380A EP3612191B1 EP 3612191 B1 EP3612191 B1 EP 3612191B1 EP 18788380 A EP18788380 A EP 18788380A EP 3612191 B1 EP3612191 B1 EP 3612191B1
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0657—Cardiomyocytes; Heart cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
- C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem cells
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
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- General Health & Medical Sciences (AREA)
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- Bioinformatics & Cheminformatics (AREA)
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- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Cell Biology (AREA)
- Epidemiology (AREA)
- Developmental Biology & Embryology (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Cardiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Vascular Medicine (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Immunology (AREA)
- Virology (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Rheumatology (AREA)
Claims (13)
- Kardiosphären-abgeleitete Zellen (CDCs) für die Verwendung bei der Behandlung von Skelettmuskeldystrophie bei einem Subjekt, wobei die Behandlung eine Verabreichung einer therapeutisch wirksamen Menge von CDCs, die zur Behandlung eines dystrophischen Skelettmuskels des Subjekts ausreicht, umfasst, wobei das Subjekt ein Mensch ist, wobei die Verabreichung intravenöse Injektion umfasst.
- CDCs für die Verwendung nach Anspruch 1, wobei die Verabreichung einer therapeutisch wirksamen Menge von CDCs über zwei oder mehr Verabreichungen erfolgt.
- CDCs für die Verwendung nach Anspruch 2, wobei die zwei oder mehr Verabreichungen von CDCs in Abständen von drei Monaten gegeben werden, um eine therapeutisch wirksame Menge von CDCs an einen Zielskelettmuskel abzugeben.
- CDCs für die Verwendung nach einem der Ansprüche 1-3, wobei die therapeutisch wirksame Menge von CDCs 150 x 106 CDCs beträgt oder mindestens beträgt.
- Zusammensetzung, die eine therapeutisch wirksame Menge von Kardiosphärenabgeleiteten Zellen (CDCs) umfasst, für die Verwendung in einem Verfahren zur Behandlung von Skelettmuskeldystrophie bei einem Subjekt, das diese benötigt, wobei die Behandlung Folgendes umfasst:Verabreichen einer ersten Dosis der Zusammensetzung an das Subjekt,
wobei die therapeutisch wirksame Menge der ersten Dosis im Bereich von 1 x 107 bis 1 x 109 CDCs liegt;Abwarten einer ersten Zeitdauer nach Verabreichung der ersten Dosis,
wobei die erste Zeitdauer zwischen 1 und 6 Monaten beträgt;Verabreichen einer zweiten Dosis einer Zusammensetzung, die eine therapeutisch wirksame Menge von CDCs umfasst, an das Subjekt,
wobei die therapeutisch wirksame Menge der zweiten Dosis im Bereich von 1 x 107 bis 1 x 109 CDCs liegt;Abwarten einer zweiten Zeitdauer nach Verabreichung der zweiten Dosis,
wobei die zweite Zeitdauer zwischen 1 und 6 Monaten beträgt;Verabreichen von mindestens einer zusätzlichen Dosis einer Zusammensetzung, die eine therapeutisch wirksame Menge von CDCs umfasst, an das Subjekt,
wobei die therapeutisch wirksame Menge der mindestens einen zusätzlichen Dosis im Bereich von 1 x 107 bis 1 x 109 CDCs liegt; undAbwarten von mindestens einer zusätzlichen Zeitdauer nach Verabreichung der mindestens einen zusätzlichen Dosis,
wobei die zweite Zeitdauer zwischen 1 und 6 Monaten beträgt;wobei die Verabreichungen zu einer Verbesserung von körperlicher Belastbarkeit oder Muskelfunktion führen,wobei die CDCs in Bezug auf das Subjekt allogen sind,wobei die Verabreichungen keine signifikante Immunantwort bei dem Subjekt induzieren,wobei die Verabreichungen systemische Verabreichung umfassen,wobei das Subjekt ein Mensch ist undwobei die Verabreichung intravenöse Injektion umfasst. - CDCs für die Verwendung nach einem der Ansprüche 1-4 oder die Zusammensetzung für die Verwendung nach Anspruch 5, wobei eine Verabreichung die Expression von einem oder mehreren Markern von T-Zell-Aktivierung oder -Proliferation verändert.
- CDCs für die Verwendung nach einem der Ansprüche 1-4 oder Anspruch 6 oder die Zusammensetzung für die Verwendung nach einem der Ansprüche 5 oder 6, wobei einer oder mehrere Marker von T-Zell-Aktivierung oder -Proliferation CD69 und HLA-DR umfassen.
- CDCs für die Verwendung nach einem der Ansprüche 1-4 oder Anspruch 6 oder 7, wobei die Verwendung Folgendes umfasst:Verabreichen von mindestens zwei Dosen einer therapeutisch wirksamen Menge von CDCs an das Subjekt;wobei die mindestens zwei Dosen 1 bis 6 Monate getrennt voneinander verabreicht werden; undwobei das Verabreichen von mindestens zwei Dosen einer therapeutisch wirksamen Menge von CDCs an das Subjekt keine signifikante Immunantwort bei dem Subjekt induziert.
- CDCs für die Verwendung nach einem der Ansprüche 1-4 oder Anspruch 6-8 oder die Zusammensetzung für die Verwendung nach einem der Ansprüche 5-7, wobei die Skelettmuskeldystrophie die Duchenne-Muskeldystrophie (DMD) umfasst, die einen dystrophischen Skelettmuskel involviert.
- CDCs für die Verwendung nach einem der Ansprüche 1-4 oder Anspruch 6-9 oder die Zusammensetzung für die Verwendung nach einem der Ansprüche 5-7 oder 9, wobei der dystrophische Skelettmuskel einen dystrophischen Skelettmuskel des Diaphragmas, des Arms oder des Beins umfasst.
- CDCs für die Verwendung nach einem der Ansprüche 1-4 oder Anspruch 6-10 oder die Zusammensetzung für die Verwendung nach einem der Ansprüche 5-7, 9 oder 10, wobei die mindestens zwei Dosen oder die erste und zweite Dosis 6 Wochen getrennt voneinander verabreicht werden.
- CDCs für die Verwendung nach einem der Ansprüche 1, 2 oder 4, wobei die therapeutisch wirksame Menge von CDCs 1 x 107 bis 1 x 109 CDCs umfasst.
- CDCs für die Verwendung nach einem der Ansprüche 1-4, 6-10 oder 12 oder die Zusammensetzung für die Verwendung nach einem der Ansprüche 5-7, 9 oder 10, wobei die CDCs allogene menschliche CDCs sind.
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP25210912.9A EP4699658A2 (de) | 2017-04-19 | 2018-04-18 | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762487393P | 2017-04-19 | 2017-04-19 | |
| US201762487402P | 2017-04-19 | 2017-04-19 | |
| US201762487408P | 2017-04-19 | 2017-04-19 | |
| US201762535672P | 2017-07-21 | 2017-07-21 | |
| US201762569440P | 2017-10-06 | 2017-10-06 | |
| US201862614753P | 2018-01-08 | 2018-01-08 | |
| PCT/US2018/028184 WO2018195210A1 (en) | 2017-04-19 | 2018-04-18 | Methods and compositions for treating skeletal muscular dystrophy |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP25210912.9A Division-Into EP4699658A2 (de) | 2017-04-19 | 2018-04-18 | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
| EP25210912.9A Division EP4699658A2 (de) | 2017-04-19 | 2018-04-18 | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| EP3612191A1 EP3612191A1 (de) | 2020-02-26 |
| EP3612191A4 EP3612191A4 (de) | 2020-12-30 |
| EP3612191B1 true EP3612191B1 (de) | 2026-03-11 |
Family
ID=63856114
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP25210912.9A Pending EP4699658A2 (de) | 2017-04-19 | 2018-04-18 | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
| EP18788380.6A Active EP3612191B1 (de) | 2017-04-19 | 2018-04-18 | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP25210912.9A Pending EP4699658A2 (de) | 2017-04-19 | 2018-04-18 | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US11759482B2 (de) |
| EP (2) | EP4699658A2 (de) |
| JP (3) | JP7336769B2 (de) |
| AU (2) | AU2018255346B2 (de) |
| CA (1) | CA3059910A1 (de) |
| WO (1) | WO2018195210A1 (de) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11660317B2 (en) | 2004-11-08 | 2023-05-30 | The Johns Hopkins University | Compositions comprising cardiosphere-derived cells for use in cell therapy |
| AU2013302799B2 (en) | 2012-08-13 | 2018-03-01 | Cedars-Sinai Medical Center | Exosomes and micro-ribonucleic acids for tissue regeneration |
| CA2962444C (en) | 2014-10-03 | 2023-09-05 | Cedars-Sinai Medical Center | Cardiosphere-derived cells and exosomes secreted by such cells in the treatment of muscular dystrophy |
| EP3402543B1 (de) | 2016-01-11 | 2021-09-08 | Cedars-Sinai Medical Center | Aus kardiosphäre stammende zellen und durch solche zellen sekretierte exosomen in der behandlung von herzversagen mit konservierter auswurffraktion |
| WO2017210652A1 (en) | 2016-06-03 | 2017-12-07 | Cedars-Sinai Medical Center | Cdc-derived exosomes for treatment of ventricular tachyarrythmias |
| US11541078B2 (en) | 2016-09-20 | 2023-01-03 | Cedars-Sinai Medical Center | Cardiosphere-derived cells and their extracellular vesicles to retard or reverse aging and age-related disorders |
| EP4699658A2 (de) * | 2017-04-19 | 2026-02-25 | Cedars-Sinai Medical Center | Verfahren und zusammensetzungen zur behandlung von skelettmuskeldystrophie |
| US11660355B2 (en) | 2017-12-20 | 2023-05-30 | Cedars-Sinai Medical Center | Engineered extracellular vesicles for enhanced tissue delivery |
| US12146137B2 (en) | 2018-02-05 | 2024-11-19 | Cedars-Sinai Medical Center | Methods for therapeutic use of exosomes and Y-RNAS |
| JP2022104813A (ja) * | 2020-12-29 | 2022-07-11 | アイ ピース,インコーポレイテッド | リプログラミング因子を導入された細胞の培養方法 |
| EP4230196A1 (de) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Verbindungen zur verwendung bei der behandlung von dystrophinopathien |
| WO2025085731A1 (en) * | 2023-10-18 | 2025-04-24 | Children's National Medical Center | TRANSFORMING GROWTH FACTOR ß (TGF-ß) OLIGONUCLEOTIDE COMBINATION THERAPY FOR TREATING MUSCULOSKELETAL DISEASES |
| WO2025122177A1 (en) * | 2023-12-05 | 2025-06-12 | Leksum LLC | Extracellular vesicles for therapy |
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- 2018-04-18 EP EP25210912.9A patent/EP4699658A2/de active Pending
- 2018-04-18 EP EP18788380.6A patent/EP3612191B1/de active Active
- 2018-04-18 AU AU2018255346A patent/AU2018255346B2/en active Active
- 2018-04-18 WO PCT/US2018/028184 patent/WO2018195210A1/en not_active Ceased
- 2018-04-18 JP JP2019556176A patent/JP7336769B2/ja active Active
- 2018-04-18 US US16/605,489 patent/US11759482B2/en active Active
- 2018-04-18 CA CA3059910A patent/CA3059910A1/en active Pending
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2023
- 2023-06-02 US US18/205,313 patent/US20230381243A1/en active Pending
- 2023-08-09 JP JP2023130177A patent/JP7724504B2/ja active Active
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2024
- 2024-07-30 AU AU2024205194A patent/AU2024205194A1/en active Pending
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- 2025-07-28 JP JP2025125726A patent/JP2025157552A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7336769B2 (ja) | 2023-09-01 |
| WO2018195210A1 (en) | 2018-10-25 |
| AU2018255346A1 (en) | 2019-11-07 |
| AU2018255346B2 (en) | 2024-05-02 |
| EP3612191A4 (de) | 2020-12-30 |
| CA3059910A1 (en) | 2018-10-25 |
| US11759482B2 (en) | 2023-09-19 |
| AU2024205194A1 (en) | 2024-08-22 |
| JP7724504B2 (ja) | 2025-08-18 |
| EP4699658A2 (de) | 2026-02-25 |
| EP3612191A1 (de) | 2020-02-26 |
| JP2020517601A (ja) | 2020-06-18 |
| US20230381243A1 (en) | 2023-11-30 |
| JP2023145784A (ja) | 2023-10-11 |
| JP2025157552A (ja) | 2025-10-15 |
| US20200121727A1 (en) | 2020-04-23 |
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